The light at the end of the tunnel gets vivid for spinal muscular atrophy: An Editorial Highlight for "Cerebrospinal fluid proteomic profiling in nusinersen-treated patients with spinal muscular atrophy" on https://doi.org/10.1111/jnc.14953

Journal of Neurochemistry
Debashis DuttaKochupurackal P Mohanakumar

Abstract

Kessler et al. in this current issue have attempted to discern biomarker(s) for spinal muscular atrophy (SMA) by assessing alterations in cerebrospinal fluid (CSF) proteomics profile. Recently, antisense oligonucleotide (nusinersen) therapy is shown to mitigate pathologies and provide behavioral improvements in patients. This Editorial highlights the study by Kessler et al on the proteomics of CSF from adult and young patients prior to, and 10 months after nusinersen intrathecal therapy. Although the study by Kessler et al. suffers from small sample size and mixed results that deterred a strong conclusion, yet is a strong case-control study that is contemporary and important to the patients, clinicians and care-takers alike. Since identifying biomarker and characterizing the pathology in SMA are imminent necessity to advance this promising therapy, the high-throughput CSF proteomics data prior and after nusinersen therapy provide possible biomarkers that may help in identification of positive responders, the disease course, efficacy of treatment, and more accurate prognosis.

References

Jun 18, 2002·Current Opinion in Cell Biology·Sergey PaushkinGideon Dreyfuss
Nov 23, 2005·Proceedings of the National Academy of Sciences of the United States of America·Karl B Shpargel, A Gregory Matera
Jun 24, 2008·Lancet·Mitchell R Lunn, Ching H Wang
Oct 13, 2016·Annual Review of Pharmacology and Toxicology·C Frank BennettRichard S Geary
May 2, 2018·Proceedings of the National Academy of Sciences of the United States of America·Jingxin WangKristen A Johnson
Jun 2, 2018·Therapeutic Advances in Neurological Disorders·Claudia D Wurster, Albert C Ludolph
Oct 12, 2018·Therapeutic Advances in Neurological Disorders·Benjamin StolteTim Hagenacker
May 30, 2019·Annals of Clinical and Translational Neurology·Basil T DarrasCharlotte J Sumner
Jun 18, 2019·Therapeutic Advances in Neurological Disorders·Claudia D WursterMarkus Otto
Jan 7, 2020·Journal of Neurochemistry·Tobias KesslerMarkus Weiler

❮ Previous
Next ❯

Related Concepts

Related Feeds

CSF & Lymphatic System

This feed focuses on Cerebral Spinal Fluid (CSF) and the lymphatic system. Discover the latest papers using imaging techniques to track CSF outflow into the lymphatic system in animal models.

Antisense Oligonucleotides: ND

This feed focuses on antisense oligonucleotide therapies such as Inotersen, Nusinursen, and Patisiran, in neurodegenerative diseases including amyotrophic lateral sclerosis.